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Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure

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ClinicalTrials.gov Identifier: NCT03589820
Recruitment Status : Enrolling by invitation
First Posted : July 18, 2018
Last Update Posted : July 18, 2018
Sponsor:
Information provided by (Responsible Party):
Liang Peng, Third Affiliated Hospital, Sun Yat-Sen University

Brief Summary:
This study is to investigate the clinical efficacy of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration in treatment of Wilson's Disease - related liver failure. 30 patients will receive treatment of plasma exchange and continuous hemodiafiltration and internal medicine. 30 patients will receive treatment of internal medicine.

Condition or disease Intervention/treatment Phase
Wilson Disease Liver Failure Other: artificial liver support system Not Applicable

Detailed Description:
Wilson's disease (WD) is an inherited disorder in which defective biliary excretion of copper leads to its accumulation, particularly in liver and brain. Presenting symptoms of liver disease can be highly variable, ranging from asymptomatic, with only biochemical abnormalities, to overt cirrhosis with all its complications. Acute liver failure (ALF) presentation is the most dramatic and may arise with catastrophic suddenness. It is considered rare, constituting only 3% of ALF cases in the pediatric ALF series in USA and associated with a high mortality reaching 95%. Patients with acute liver failure due to WD require liver transplantation, which is lifesaving. Kido J et al reported clinical outcomes of patients with liver failure were improved by artificial liver support system using combination of plasma exchange and continuous hemodiafiltration. So the investigators design this protocol: 30 patients will receive treatment of plasma exchange and continuous hemodiafiltration and internal medicine, 30 patients will receive treatment of internal medicine.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Clinical Efficacy of Artificial Liver Support System Using Combination of Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease - Related Liver Failure
Actual Study Start Date : June 1, 2018
Estimated Primary Completion Date : June 28, 2020
Estimated Study Completion Date : July 31, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Wilson Disease

Arm Intervention/treatment
Active Comparator: Artificial liver support system group
30 patients will receive treatment of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration and internal medicine.
Other: artificial liver support system
Patients will receive the treatment of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration and internal medicine. The volume of fresh frozen plasma used in plasma exchange is 2000 millilitre. The time for continuous hemodiafiltration is about 8 hours.

No Intervention: Control group
30 patients will receive treatment of internal medicine.



Primary Outcome Measures :
  1. survival rate [ Time Frame: 48 weeks ]
    Whether patients will survive is observed in the follow-up.


Secondary Outcome Measures :
  1. liver function improvement [ Time Frame: 48 weeks ]
    Symptoms (i.e. fatigue, appetite, nausea, vomiting, jaundice, consciousness) and laboratory tests (i.e. blood cells, alanine transaminase, total bilirubin, prothrombin time, creatinine, ceruloplasmin, serum copper) are observed in the follow-up.



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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Clinical diagnosis of Wilson Disease: serum ceruloplasmin < 0.2g/L, 24 hours urinary copper > 40μg, Kayser-Fleischer ring present;
  2. Do not receive drugs to promote the excretion of copper ever before;
  3. Serum total bilirubin > 10 times upper limit of normal, prothrombin time activity < 40% or prothrombin time international ratio > 1.5.

Exclusion Criteria:

  1. Other active liver diseases;
  2. Hepatocellular carcinoma or other malignancy;
  3. Pregnancy or lactation;
  4. Human immunodeficiency virus infection or congenital immune deficiency diseases;
  5. Severe diabetes, autoimmune diseases;
  6. Other important organ dysfunctions.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03589820


Locations
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China, Guangdong
The Third Affiliated Hospital of Sun Yat-sen University
Guangzhou, Guangdong, China, 510630
Sponsors and Collaborators
Third Affiliated Hospital, Sun Yat-Sen University
Investigators
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Principal Investigator: Liang Peng, Doctor Third Affiliated Hospital, Sun Yat-Sen University

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Responsible Party: Liang Peng, Professor, Third Affiliated Hospital, Sun Yat-Sen University
ClinicalTrials.gov Identifier: NCT03589820     History of Changes
Other Study ID Numbers: XWX1
First Posted: July 18, 2018    Key Record Dates
Last Update Posted: July 18, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Liang Peng, Third Affiliated Hospital, Sun Yat-Sen University:
Wilson Disease
Liver Failure
plasma exchange
continuous hemodiafiltration

Additional relevant MeSH terms:
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Liver Failure
Liver Diseases
Liver Extracts
Hepatic Insufficiency
Hepatolenticular Degeneration
Digestive System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Metabolic Diseases
Hematinics